The United States of America, As Represented by the Secretary, Department of Health and Human Services

University of Kansas

Ellen Sidransky

Wei Ping Zheng

Juan Jose Marugan

Noel Terrence Southall

Samarjit Patnaik

Wendy Westbroek

Ehud Goldin

Omid Motabar

Frank John Schoenen

Steven Andrew Rogers

Elma Aflaki

Wei Zheng

Wendy Westbrook

Noel Southall

M Ehud Goldin

Substituted pyrazolopyrimidines and dihydropyrazolopyrimidines and related compounds, their methods of manufacture, compositions containing these compounds, and methods of use of these compounds in treating lysosomal storage disorders such as Gaucher disease are described herein. The compounds are of general Formula (I) in which variables R-Rand X are described in the application.

Substituted pyrazolopyrimidines as glucocerebrosidase activators

Compounds of Formula (I) and the pharmaceutically acceptable salts thereof are disclosed. The variables. R-R, m, n, o, and p are disclosed herein. The compounds are useful for treating Gaucher disease and inhibiting the onset of Gaucher disease symptoms in a patient having a GBA gene mutation and for treating Parkinson's disease. Pharmaceutical compositions containing compounds of Formula (I) and methods of treatment comprising administering compounds of Formula (I) are also disclosed. 

Salicylic acid derivatives useful as glucocerebrosidase activators

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Bethesda, MD, United States of America

Ellen Sidransky